FDA Approves HCV Drugs for Ages 12-17

First pediatric approval for direct-acting agents

Two direct-acting antiviral treatments for hepatitis C (HCV) can now be used to treat older children and teens, the FDA said.

The agency approved supplemental applications for sofosbuvir (Sovaldi) and the combination of ledipasvir and sofosbuvir (Harvoni) to treat chronic HCV in children ages 12 to 17, making them the first direct-acting antivirals (DAAs) treatments approved for children and adolescents.

Both treatments had previously been approved for adults.

The approvals address "an unmet need in children and adolescents," according to Edward Cox, MD, of the FDA's Center for Drug Evaluation and Research, a sentiment echoed by Karen Murray, MD, of the University of Washington School of Medicine in Seattle.

The approvals "will enable adolescents to finally benefit from interferon-free treatment for HCV infection," Murray said in a statement issued by drugmaker Gilead Sciences of Foster City, Calif.

"These therapies ... represent an important advance for HCV-infected adolescents," said Murray, who is to present related data at the International Liver Congress later this month in Amsterdam.

Harvoni was approved specifically for pediatric patients 12 or older or weighing at least 77 pounds with HCV genotypes 1, 4, 5, or 6 with no more than mild cirrhosis. To treat genotypes 2 and 3 in the same patient group, the agency approved sofosbuvir with ribavirin.

The CDC estimates that between 23,000 to 46,000 children in the U.S. have chronic HCV, most of them infected at birth.

The approvals were supported by data from two open-label trials, which showed that safety, pharmacokinetics, and efficacy of the treatments were comparable to what has been observed in adults, the FDA statement said.

In particular, more than 97% of participants in each study were able to clear the virus after 12 weeks of treatment and to remain clear by 12 weeks after follow-up -- a sustained virologic response that is regarded as a cure.

The most common adverse effects associated with Harvoni were fatigue, headache, and asthenia, the Gilead statement said, while patients treated with the sofosbuvir/ribavirin combination most commonly reported fatigue, headache, and nausea.

The FDA statement noted that, in adult patients co-infected with both hepatitis B and C, reactivation of HBV has been seen during or after treatment with DAAs, something that can result in serious liver problems or death.